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Appendix B
Speaker Biographical Sketches
Christopher P. Austin, M.D., is scientific director at the National Institutes
of Health (NIH) Center for Translational Therapeutics (NCTT), and direc-
tor of the Division of Preclinical Innovation at the National Center for
Advancing Translational Sciences, U.S. NIH. The NCTT's mission is both
to translate basic science discoveries into new treatments, particularly for
rare and neglected diseases, and to develop new technologies and para-
digms by which therapeutic development is done. The NCTT's programs
span the spectrum of translational science from RNAi biology to small
molecule probe discovery to drug development, including genome-wide
RNAi, the NIH Chemical Genomics Center (NCGC), the Therapeutics for
Rare and Neglected Diseases (TRND) program, and the Bridging Interven-
tional Development Gaps (formerly the NIH-RAID) program. The NCGC
is an ultra-high-throughput screening, informatics, and chemistry center
that profiles small molecule libraries for biological activity using its quan-
titative high-throughput screening (qHTS) technology and develops novel
compounds as probes of biology and starting points for the development of
new drugs for rare and neglected diseases. The NCGC is a partner with the
National Toxicology Project, the Environmental Protection Agency, and the
Food and Drug Administration in the Toxicology in the 21st Century Pro-
gram, which is developing in vitro signatures for in vivo toxicity endpoints.
The TRND program develops small molecules and biologics from lead to
clinical proof of concept for rare and neglected diseases. Before joining NIH
in 2002, Dr. Austin directed research programs in genomics-based target
discovery, pharmacogenomics, and neuro psychiatric drug development at
Merck, with a particular focus on schizophrenia. Dr. Austin received his
65
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66 GENOME-BASED THERAPEUTICS
A.B. in biology summa cum laude from Princeton and his M.D. from Har-
vard Medical School. He completed clinical training in internal medicine
and neurology at the Massachusetts General Hospital and finished a post-
doctoral fellowship in genetics at Harvard.
Wylie Burke, M.D., Ph.D., is professor and chair of the Department of
Bioethics and Humanities at the University of Washington. She received
a Ph.D. in genetics and an M.D. from the University of Washington and
completed a residency in internal medicine at the University of Washington.
She was a medical genetics fellow at the University of Washington from
1981 to 1982. Dr. Burke was a member of the Department of Medicine
at the University of Washington from 1983 to 2000, where she served as
associate director of the internal medicine residency program and founding
director of the University of Washington's Women's Health Care Center.
She was appointed chair of the Department of Medical History (now the
Department of Bioethics and Humanities) in October 2000. She is also
an adjunct professor of medicine and epidemiology and a member of the
Fred Hutchinson Cancer Research Center. She is a member of the Institute
of Medicine and the Association of American Physicians and is a past
president of the American Society of Human Genetics. Dr. Burke's research
addresses the social, ethical, and policy implications of genetics, including
responsible conduct of genetic and genomic research, genetic test evalua-
tion, and implications of genomic health care for underserved populations.
She is director of the University of Washington Center for Genomics and
Healthcare Equality, a National Human Genome Research Institute center
of excellence in ethical, legal, and social implications research, and co-
director of the Northwest-Alaska Pharmacogenomic Research Network.
Walter Capone, M.B.A., is the chief operating officer of the Multiple Myeloma
Research Foundation (MMRF) and the Multiple Myeloma Research Consor-
tium (MMRC). He oversees the core business operations of the MMRF and
as part of the executive committee executes the growth initiatives outlined
in the organization's strategic plan. He has 20 years of pharmaceutical and
biotechnology leadership experience in the areas of commercial develop-
ment, operations, finance, marketing, and sales in the United States and
internationally. Prior to joining the MMRF, he was the vice president of
commercial development and operations at Progenics Pharmaceuticals. He
previously worked at a number of entrepreneurial pharmaceutical and bio-
technology ventures throughout the United States and Europe including
Trimeris, T riangle Pharmaceuticals, and Cyanamid Benelux. He started his
career at leading global pharmaceutical companies including Lederle, Wyeth,
and Bristol-Myers Squibb. He received his B.A. in international relations
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APPENDIX B 67
from Brown University and he has an M.B.A. in finance and international
business from Columbia University Business School.
Nicholas Davies, Ph.D., is a partner in the pharmaceutical and life sciences
practice at PricewaterhouseCoopers (PwC), responsible for pharmaceuti-
cal and research and development strategy. He has been a key figure in
the pharmaceutical life sciences consulting and pharmaceutical industry
for over 16 years and possesses extensive knowledge spanning research,
development, commercial and marketing, and the external payer, access,
and regulator environments. Prior to joining PwC, Dr. Davies was respon-
sible for global research and development strategy at Pfizer and led a
number of key research and development transformations, integrations,
and mergers. As a former PwC management consultant and IBM business
consulting research and development leader, he led and delivered complex
engagements in major mergers and acquisitions, extensive restructuring and
downsizing, research and development productivity projects, personalized
medicine and diagnostics strategies, portfolio and decision making strate-
gies, outsourcing, clinical research organization and partnering strategies,
operational strategies in discovery and clinical development, enterprise risk
management, Asia and emerging market strategies, out- and in-licensing,
and commercial assessments of pipeline assets. He has also led research
laboratories and departments at Novartis and AstraZeneca. Dr. Davies
gained his Ph.D. in immunology and genetics at Cambridge University, UK.
Radoje Drmanac, Ph.D., is a co-founder of Complete Genomics and has
served as chief scientific officer since July 2005. In 2001, Dr. Drmanac co-
founded Callida Genomics, Inc., a DNA sequencing company, and served as
Callida's chief scientific officer from 2001 to 2004 and as its president since
2004. In 1994, Dr. Drmanac co-founded Hyseq, Inc., a DNA array technol-
ogy company that became Hyseq Pharmaceuticals, Inc. and later merged
with Variagenics, Inc. to become Nuvelo, Inc., and served as its senior vice
president of research from 1994 to 1998 and as its chief scientific officer
from 1998 to 2001. Prior to that, Dr. Drmanac served as a group leader
at Argonne National Laboratory. Dr. Drmanac received a B.S., M.S., and
Ph.D. in molecular biology from the University of Belgrade.
Deborah Dunsire, M.D., has been the president and chief executive officer of
Millennium Pharmaceuticals Inc., since July 2005. In 2008 Millennium was
acquired by Takeda Pharmaceuticals of Japan, becoming the global oncol-
ogy center of excellence for Takeda under her leadership. Dr. Dunsire joined
Millennium from her role as senior vice president and North A
merican
region head of the oncology business unit of Novartis. Prior to her move to
the United States in 1994, Dr. Dunsire worked in the global headquarters
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68 GENOME-BASED THERAPEUTICS
of Sandoz in Switzerland managing launch and growth of global products
in the field of immunology and dermatology. She joined Sandoz in South
Africa in 1988 as a clinical researcher and expanded her résumé to include
portfolio and specialty market management. Dr. Dunsire is a member of
the board of directors of Allergan Inc. and serves as a director of the Bio-
technology Industry Organization where she co-chairs the Committee on
Reimbursement and serves as a member of the executive committee. Her
not-for-profit board memberships include CancerCare Inc.; the Gabrielle's
Angel Foundation for Cancer Research; and the Boston Museum of Science,
where she chairs the investment review board; and she is also a member of
the Massachusetts General Hospital research advisory council. She served
as a director of the Pharmaceutical Research and Manufacturers of America
from 2005 to 2008 and as a director of the California Healthcare Institute
from 2002 to 2005. Dr. Dunsire was the 2001 recipient of the American
Cancer Society Excalibur Award, the 2000 recipient of the Health Care Busi-
ness Women's Association Rising Star Award, the 2009 Health Care Business
Women's Association Woman of the Year Award, the 2011 Massachusetts
Biotechnology Organization's Innovative Leadership Award, and the 2011
Golden Door Award from the International Institute of New England. She
received a Ph.D. honoris causa from Worcester Polytechnic Institute in 2007.
Dr. Dunsire graduated as a physician from the University of Witwatersrand
in Johannesburg, South Africa.
Victor J. Dzau, M.D., is the chancellor for health affairs and James B. Duke
Professor of Medicine at Duke University and the president and chief execu-
tive officer of Duke University Health System. Dr. Dzau was previously
the Hersey Professor of Theory and Practice of Medicine and chairman
of medicine at Harvard Medical School's Brigham and Women's Hospital
and the chairman of the department of medicine at Stanford University.
Dr. Dzau has made a significant impact on medicine through his seminal
research in cardiovascular medicine, his pioneering work in the discipline
of vascular medicine, and recently his leadership in health care innovation.
His important work on the renin angiotensin system (RAS) paved the way
for the contemporary understanding of RAS in cardiovascular disease and
the development of RAS inhibitors as therapeutics. Dr. Dzau also pioneered
gene therapy for vascular disease and was the first to introduce DNA decoy
molecules to block transcription as gene therapy in vivo. Recently his semi-
nal work on stem cell "paracrine mechanism" provided novel insights into
stem cell biology and therapy. As a leader of academic medicine, Dr. Dzau's
vision is that academic health centers must lead the transformation through
innovation, translation, and globalization. To achieve this vision he has
established the Duke Translational Medicine Institute, Duke Global Health
Institute, Duke Initiative in Health Innovation, and Duke National Univer-
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APPENDIX B 69
sity of Singapore Graduate Medical School in Singapore. Among his honors
and recognitions are the prestigious Gustav Nylin Medal from the S wedish
Royal College of Medicine; the Max Delbruck Medal from Humboldt Uni-
versity, Charite and Max Planck Institute; the Commemorative Gold Medal
from Ludwig Maximillian University of Munich and Frey-Werle Founda-
tion; the Inaugural Hatter Award from the Medical Research Council of
South Africa; the Polzer Prize from the European Society of Sciences and
Arts; the Ellis Island Medal of Honor of the USA; the Novartis Award for
Hypertension Research; the Distinguished Scientist Award from the Ameri-
can Heart Association (AHA); and the 2010 AHA Research Achievement
Award for his contributions to cardiovascular biology and medicine. He has
received six honorary doctorates. He serves on the council of the Institute
of Medicine of the National Academy of Sciences, the board of directors
of Research America, and the board of health governors of the World
Economic Forum. He is also board chair of the Association of Academic
Health Centers. He has chaired the NIH Cardiovascular Disease Advisory
Committee as well as the Council of Atherosclerosis, Thrombosis and Vas-
cular Biology of the AHA, and has served on the advisory council to the
director of NIH.
Garret A. FitzGerald, M.D., FAHA, is the McNeil Professor in Translational
Medicine and Therapeutics at the University of Pennsylvania in Philadelphia,
where he chairs the Department of Pharmacology and directs the Insti-
tute for Translational Medicine and Therapeutics (ITMAT). Dr. FitzGerald
trained in medicine at University College Dublin and its teaching hospitals
and in statistics at Trinity College in Dublin and the London School of
Hygiene. Following fellowships at the Royal Postgraduate Medical School
in London, the Max Planck Institute in Cologne, and Vanderbilt University,
Dr. FitzGerald joined the faculty at Vanderbilt and eventually led the Divi-
sion of Clinical Pharmacology as the William Stokes Professor of Experi-
mental Therapeutics. He moved in 1991 to lead the Department of Medicine
and Experimental Therapeutics at University College, Dublin, and then
returned in 1994 to the United States to take up direction of the Center
for Experimental Therapeutics and the General Clinical Research Center as
the Robinette Professor of Cardiovascular Medicine at Penn. These struc-
tures were subsumed into ITMAT when it was founded in 2004, antici-
pating the funding of clinical and translational research centers 2 years later
by the NIH. ITMAT has grown to more than 650 members and supports
research programs, faculty recruitment, education, and infrastructural devel-
opments relevant to translational research. Dr. FitzGerald has served as chair
of the Department of Pharmacology at Penn. The department is routinely
placed in the top three in NIH funding in the United States and supports a
graduate group in pharmacological sciences with about 90 students.
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70 GENOME-BASED THERAPEUTICS
Dr. FitzGerald's research has been characterized by an integrative
approach to elucidating the mechanisms of drug action, drawing on work in
cells, model organisms, and humans. His work contributed substantially to
the development of low-dose aspirin. He was the first to describe the dose-
dependent suppression by aspirin of thromboxane and p rostacyclin bio
synthesis in vivo and to discover that inhibition of platelet c
yclooxygenase
by low-dose aspirin occurred in the presystemic circulation and to char-
acterize the interaction by which non-steroidal anti- inflammatory drugs
(NSAIDs) like ibuprofen could interact with and undermine cardio
protection from aspirin. Dr. FitzGerald's group was the first to predict
and then mechanistically explain the cardiovascular hazard from NSAIDs.
Since his first prediction of a potential hazard, based on clinical pharma-
cological studies 12 years ago, evidence consistent with the mechanism
proposed-- suppression of COX-2 derived prostacyclin--has emerged from
multiple studies in model systems, including many genetically manipu-
lated mice created by his group; human genetics; randomized comparisons
amongst NSAIDs; and seven placebo-controlled trials of three structurally
distinct NSAIDs designed to be specific for inhibition of COX-2. Aside
from this work, Dr. FitzGerald has also discovered many products of lipid
peroxidation and established their utility as indices of oxidant stress in
vivo. Using this methodology he demonstrated that conventional doses of
vitamins E and C have no impact on lipid peroxidation in healthy individu-
als with intact endogenous antioxidant defense, that social consumption of
alcohol has a pro-oxidant effect, and that suppression of lipid peroxidation
retards atherogenesis in mice. His laboratory was the first to discover a
molecular clock in the cardiovascular system and has contributed substan-
tially to our understanding of the importance of peripheral clocks in the
regulation of cardiovascular and metabolic function.
Dr. FitzGerald's papers have been published in journals such as Cell,
Science, Nature, the New England Journal of Medicine, the Lancet, JAMA,
PNAS, JCI, and Nature Medicine and have been cited more than 30,000
times. He has also published on science policy in the lay and profes-
sional press and is an occasional sports commentator for Il Manifesto. He
has been awarded honorary degrees from University College Dublin and
the Universities of Edinburgh and Frankfurt. Among his awards are the
Harvey Medal, the Boyle Medal, the Taylor Prize, and the Cameron Prize.
Dr. FitzGerald serves on the peer review advisory committee of the NIH, the
science board of the FDA, and the drug forum of the Institute of Medicine.
Jane Fridlyand, Ph.D., is a senior statistical scientist in the Department
of Biostatistics at Genentech/Roche. Dr. Fridlyand received her Ph.D. in
statistics in 2001 from the University of California, Berkeley; her disser-
tation was focused on the applications of statistics to high-dimensional
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APPENDIX B 71
biological data, including sequencing, genotyping, and early expression
microarrays. She continued on to a postdoctoral position at the University
of California, San Francisco (UCSF) Cancer Center where she developed
novel methods for the analyses of genome-wide copy-number data. In 2003
she transitioned to a faculty position at the Department of Epidemiology
and Biostatistics at UCSF. Dr. Fridlyand's main area of research included
development of new approaches to the integration of different modalities
of high-dimensional genomic and genetic data in cancer with the aim of
identifying novel tumor subtypes relevant to disease etiology and prognosis.
Dr. Fridlyand has co-authored more than 50 peer-reviewed publications
and multiple book chapters, was a key contributor to a number of funded
NIH applications, and has been an invited speaker at many national and
international meetings. In 2007, Dr. Fridlyand joined early clinical develop-
ment, oncology, at Genentech. In the past 5 years, her work has focused on
developing strategies for incorporation of biomarkers into clinical develop-
ment programs. Currently she leads global biometrics efforts at Roche in
personalized health care.
Felix W. Frueh, Ph.D., is president of the Medco Research Institute, lead-
ing Medco's real-world, outcomes-based research in personalized medicine.
Dr. Frueh was associate director for genomics at the FDA and managing
partner at Stepoutside Consulting and held senior positions at Transgenomic
and Protogene Laboratories. He is a member of the board of the Person-
alized Medicine Coalition and TcLand Expression, Inc. and is adjunct
faculty at the Institute for Pharmacogenomics and Individualized Therapy
at the University of North Carolina. Dr. Frueh held faculty appointments in
the departments of pharmacology and medicine at Georgetown University
in Washington, DC, and was a fellow at Stanford University and the Univer-
sity of Basel, Switzerland, where he also received his Ph.D. in biochemistry.
Geoffrey Ginsburg, M.D., Ph.D., is the founding director for Genomic
Medicine at Duke University and assumed his current position in the Duke
Institute for Genome Sciences & Policy in 2004. He is also the founding
executive director of the Center for Personalized Medicine established in the
Duke University Health System in 2010. Dr. Ginsburg is currently professor
of medicine and pathology at Duke University Medical Center. While at
Duke, Dr. Ginsburg has pioneered translational genomics, initiating pro-
grams in genome enabled biomarker discovery, longitudinal registries with
linked molecular and clinical data, biomarker-informed clinical trials, and
the development of novel practice models and implementation research for
the integration of genomic tools in heath care systems. With a strong com-
mitment to interdisciplinary science he has led projects to develop predictive
models for common complex diseases using high-dimensional genomic data
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72 GENOME-BASED THERAPEUTICS
as well as collaborations with engineering groups to develop novel point
of care sensors.
Dr. Ginsburg's work spans oncology, infectious diseases, cardiovascular
disease and metabolic disorders. His research is addressing the challenges
for translating genomic information into medical practice using new and
innovative paradigms and the integration of personalized medicine into
health care. He is an internationally recognized expert in genomics and
personalized medicine with over 200 published papers and funding from
NIH, DOD, DARPA, the Gates Foundation, and industry. In 1990, he
joined the faculty of Harvard Medical School, where he was director of
Preventive Cardiology at Beth Israel Hospital and led a laboratory in
applied genetics of cardiovascular disease at Children's Hospital. In 1997
he joined M illennium Pharmaceuticals Inc. as senior program director for
cardiovascular diseases and was eventually appointed vice president of
Molecular and Personalized Medicine, where he was responsible for devel-
oping pharmacogenomic strategies for therapeutics, as well as biomarkers
for disease and their implementation in the drug development process. He
has received a number of awards for his research accomplishments, includ-
ing the Innovator in Medicine Award from Millennium in 2004 and the
Basic Research Achievement Award in Cardiovascular Medicine from Duke
University in 2005. He is a founding member and former board member
of the Personalized Medicine Coalition, a senior consulting editor for the
Journal of the American College of Cardiology, an editor for the HUGO
Journal, and an editorial advisor for Science Translational Medicine. In
addition he is the editor of Genomic and Personalized Medicine (Elsevier)
whose first edition was published in 2009.
Dr. Ginsburg has been a member of the Secretary of Veterans Affairs
Advisory Council on Genomic Medicine and the National Advisory Coun-
cil for Human Genome Research at NIH. He is currently an international
expert panel member for Genome Canada, a member of the Board of
External Experts for the National Heart, Lung, and Blood Institute, the
Institute of Medicine's Roundtable on Translating Genomic-Based Research
for Health, and a member of the External Scientific Panel for the Pharmaco
genomics Research Network. Dr. Ginsburg has recently been appointed to
the Advisory Council for the newly established National Center for Advanc-
ing Translational Sciences at NIH and has recently been nominated to serve
on the World Economics Forum's Global Agenda Council on Personalized
and Precision Medicine. Dr. Ginsburg received his M.D. and Ph.D. in
biophysics from Boston University and completed an internal medicine
residency at Beth Israel Hospital in Boston, Massachusetts. Subsequently,
he pursued postdoctoral training in clinical cardiovascular medicine at Beth
Israel Hospital and in molecular biology at Children's Hospital as a Bugher
Foundation Fellow of the American Heart Association.
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APPENDIX B 73
Margaret A. Hamburg, M.D., is the 21st commissioner of the U.S. Food
and Drug Administration (FDA). As the top official at the FDA, Dr. Ham-
burg is committed to strengthening programs and policies that enable the
agency to carry out its fundamental mission--to protect and promote
the public health. Only the second woman ever to serve as commissioner,
Dr. Hamburg earned her M.D. from Harvard Medical School and com-
pleted her residency at what is now New York Presbyterian HospitalWeill
Cornell Medical Center. She conducted neuroscience research at Rockefeller
University in New York and at the National Institute of Mental Health
and later focused on AIDS research as assistant director of the National
Institute of Allergy and Infectious Diseases. In 1991, after just a year in the
New York City Department of Health, Dr. Hamburg was named its com-
missioner. During her 6-year tenure she implemented rigorous public health
initiatives that tackled the city's most pressing crises head-on, including
improved services for women and children, a needle-exchange program to
combat HIV transmission, and the nation's first public health bioterrorism
defense program. The most celebrated achievement during her leadership
was her aggressive approach to the city's tuberculosis epidemic, which led to
an 86 percent decline in drug-resistant TB in just 5 years. In 1997, 3 years
after she was elected one of the youngest-ever members of the Institute of
Medicine, President Bill Clinton named Dr. Hamburg assistant secretary
for planning and evaluation in the U.S. Department of Health and Human
Services, where she served until the end of the Clinton administration. She
then became founding vice president for biological programs at the Nuclear
Threat Initiative, a foundation dedicated to reducing the threat to public
safety from nuclear, chemical, and biological weapons. President Barack
Obama nominated Dr. Hamburg for the post of FDA commissioner on
March 14, 2009. As the commissioner of food and drugs, Dr. Hamburg has
emphasized the critical role of innovation in meeting the nation's rapidly
growing public health needs. She provided leadership for the implementa-
tion of three groundbreaking measures: the Family Smoking Prevention and
Tobacco Control Act, a 2009 law that gives FDA the authority to regulate
the manufacture, distribution, and marketing of tobacco products; the Food
Safety Modernization Act of 2011, which changed the focus of food safety
measures from responding to food-borne outbreaks of illness to preventing
them; and a thorough review of the system for the evaluation and approval
of medical devices. Beyond these specific undertakings, Dr. Hamburg has set
the agency's paramount course for fulfilling two central public health tasks.
She has launched a nationwide public-private effort to strengthen regula-
tory science as a means for advancing the development and evaluation of
innovative, breakthrough medical products, and she is leading FDA's trans-
formation into a global regulatory agency capable of ensuring the safety
and quality of imported food, drugs, and medical devices. Commissioner
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74 GENOME-BASED THERAPEUTICS
Hamburg is committed to ensuring that FDA is poised to meet the public
health challenges of the 21st century.
Steffan N. Ho, M.D., Ph.D., is currently director of translational oncol-
ogy at Pfizer Inc. He received a Ph.D. in immunology and an M.D. from
the Mayo Clinic. He completed a residency in pathology at the Stanford
University Medical Center and a postdoctoral fellowship in the Howard
Hughes Medical Institute, also at Stanford. Dr. Ho was on the faculty at the
University of California, San Diego, School of Medicine in the Department
of Pathology and the Department of Cellular and Molecular Medicine. He
subsequently led the development of the translational oncology group at
Biogen Idec. In his current position at Pfizer, Dr. Ho plays a leadership role
in integrating translational research efforts with experimental medicine
strategies to define mechanism of action, understand pharmacodynamic
relationships, identify rational therapeutic combinations, and investigate
predictive biomarker hypotheses. He has focused on integrating drug and
diagnostic development strategies, including the coordination of strategic
collaborations supporting predictive diagnostic test development. He also
functions as the translational oncology lead for the Xalkori program.
Kathy L. Hudson, Ph.D., is the deputy director for science, outreach, and
policy at the National Institutes of Health (NIH) where she oversees the
activities of the associate directors for communications and public liaison,
legislative policy and analysis, and science policy. In addition, Dr. Hudson
works with NIH leadership to develop and implement new strategic and
scientific initiatives and is the NIH liaison with the U.S. Department of
Health and Human Services. She also represents the NIH--and the NIH
director--in high-level collaborations and negotiations with other federal
agencies, such as FDA, Centers for Disease Control and Prevention, and
the White House Office of Science and Technology Policy, as well as with
private research institutions, patient voluntary organizations, and profes-
sional societies. In addition to her role in the Office of the Director at
NIH, in December 2011 Dr. Hudson became the acting deputy director of
the new National Center for Advancing Translational Sciences (NCATS)
at NIH. She also serves as the acting director of the Office of Strategic
Communications, Alliances, and Policy at NCATS. Dr. Hudson holds a
Ph.D. in molecular biology from the University of California, Berkeley, an
M.S. in microbiology from the University of Chicago, and a B.A. in biology
from Carleton College.
Thomas Lehner, Ph.D., M.P.H., is the director of the Office for G
enomics
Research Coordination and chief, Genomics Research Branch at the
National Institute of Mental Health (NIMH), National Institutes of Health
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APPENDIX B 75
(NIH). He oversees and coordinates all efforts associated with genomics
research for NIMH and is the principal advisor to the NIMH director
and the NIMH scientific director for issues related to genetics and genom-
ics. A native of Vienna, Austria, he received a Ph.D. in genetics from
the University of Vienna and an M.P.H. in epidemiology from Columbia
University. He completed his doctoral training in the laboratory of Jurg
Ott at Columbia University and later moved to the Rockefeller University
where he worked with Jeff Friedman as the Associate Director of the Starr
Center for Human Genetics and as senior research scientist affiliated with
the Laboratory for Statistical Genetics. He has also served as an infectious
disease epidemiologist for the City of New York and as director of science
and research for a subsidiary of Millennium Pharmaceuticals. Since joining
NIMH in 2004, Thomas has been instrumental in expanding the NIMH
genomics portfolio and the NIMH Repository at Rutgers University while
also promoting the team science approach in genomics by forging interna-
tional collaborative efforts and consortia.
Peter Mueller, Ph.D., joined Vertex in July 2003. As executive vice presi-
dent, global research and development, and chief scientific officer, he pro-
vides strategic oversight for Vertex's worldwide drug discovery research
programs, pharmaceutical development, quality assurance and control,
and pharmaceutical operations as well as clinical and nonclinical develop-
ment, regulatory, and medical affairs. Key areas of Vertex's research and
development are hepatitis C (HepC), cystic fibrosis (CF), immune-mediated
inflammatory diseases (IMIDs), cancer, and neurological diseases, which
led in 2011 to the successful approval and launch of INCIVEK (HepC), a
NDA/MAA submission for KALYDECO (CF) with FDA approval in Janu-
ary 2012 and several proof of clinical concept candidates in various disease
areas. Prior to coming to Vertex, Dr. Mueller served as senior vice president,
research and development, for Boehringer Ingelheim Pharmaceuticals, Inc.,
where he was responsible for the development of all drug candidates of the
company's worldwide portfolio in North and South America, Canada, and
Japan, beginning in 1997. He also led research programs in the areas of
immunology, inflammation, cardiovascular disease, and gene therapy on a
global basis. During his time with Boehringer Ingelheim (BI), Dr. Mueller
oversaw the discovery of numerous development candidates, held several
positions in basic research, medicinal chemistry, and management in dif-
ferent centers of BI worldwide. Dr. Mueller received both an undergradu-
ate degree and a Ph.D. in chemistry at the Albert Einstein University of
Ulm, Germany, where he also holds a professorship in theoretical organic
chemistry. He completed fellowships in quantum pharmacology at Oxford
University and in biophysics at Rochester University. Special fields of study
are synthetic organic chemistry, computational chemistry (cheminformat-
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76 GENOME-BASED THERAPEUTICS
ics and bioinformatics), RNA-biophysics, atherosclerosis research, IMIDs,
neurodegenerative diseases, infection, oncology, gene/epigenetic technology,
and management strategies. He is a board member of various scientific
and political societies, such as the Gesellschaft Deutscher Chemiker and
Verband Chemische Industrie; the Royal Society of Chemistry; the U.S.-
India Chamber of Commerce Biotech, Pharma & Medical Devices Council;
the Industrial Research Institute; RNA-the Society; the Association of Stra-
tegic Alliance Professionals; the American Association for the Advancement
of Science; and the Harvard Accelerator Fund. Before he left Connecticut to
join Vertex, Dr. Mueller was also a member of Governor Roland's Council
on Economic Competitiveness and Technology for the State of Connecticut.
Laura K. Nisenbaum, Ph.D., is senior research advisor in translational
medicine and tailored therapeutics at Eli Lilly and Company. She received
a Ph.D. in neuroscience from the University of Pittsburgh in 1991. Prior
to this, Dr. Nisenbaum received a Fulbright Scholarship to study neuro
science in Cologne, Germany. Before joining Lilly, she completed post-
doctoral fellow ships at the National Institute of Mental Health and the
University of Tennessee College of Medicine. In addition she was an assis-
tant p
rofessor in the Department of Physiology and Neurobiology at the
University of C
onnecticut from 1995 to 1998. Dr. Nisenbaum joined Lilly
in 1998 and while there she has made significant contributions to drug dis-
covery and development for the treatment of psychiatric and neurological
disorders, especially schizophrenia. She has developed and implemented
molecular profiling methodologies for novel target validation and bio-
marker development. In addition, Dr. Nisenbaum has applied pharmaco
genomics across the neuroscience drug development portfolio to help tailor
Lilly drugs for the right patient, leading to improved individual patient
outcomes.
Michael A. Pacanowski, Pharm.D., M.P.H., is a clinical pharmacologist
and team leader of the genomics group in the Office of Clinical Phar-
macology at the FDA. Dr. Pacanowski received his Pharm.D. from the
Philadelphia College of Pharmacy. He then completed clinical training at
Bassett Healthcare in Cooperstown, New York, and a clinical research
fellow ship in cardio vascular pharmacogenomics at the University of
Florida, where he also received his M.P.H. Dr. Pacanowski's expertise is
in the area of genetic epidemiology and public health genomics, specifi-
cally as related to pharmaco genomic strategies in drug development and
utilization. At the FDA, he oversees review of investigational and new drug
applications, contributes to regulatory policy development, and conducts
research that supports FDA's core public health mission.
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Gary Palmer, M.D., J.D., M.B.A., M.P.H., is a medical oncologist with a
career spanning three decades in oncology, initially as a clinician in both the
academic and community settings and then as a biotech industry executive
with diagnostic and therapeutic experience. Currently, he is the senior vice
president of medical affairs and commercial development at Foundation
Medicine. Dr. Palmer joined Foundation Medicine from On-Q-ity, where
he was chief medical officer and head of development for DNA repair
marker development and circulating tumor cell technology. He also served
as vice president of medical affairs at Genomic Health, Inc., where he was
instrumental in the commercialization of the Oncotype DX breast cancer
assay. Prior to Dr. Palmer's tenure with Genomic Health, he held leadership
positions at Kosan Biosciences and Salmedix, Inc. He also spent 5 years
at Amgen, Inc., where he was involved in the clinical development and
commercialization of Neupogen, Neulasla, and Aranesp. Prior to joining
industry, he served as director of the Medical Breast Service at the Univer-
sity of California, Davis, Cancer Center and chief of medical oncology at
Mercy Health System, Sacramento. Dr. Palmer received a B.A. from Yale
University and an M.D. from the Stanford University School of Medicine.
He completed his internal medicine training at the Boston City Hospital
and his oncology fellowship at the Massachusetts General Hospital. He
also holds an M.B.A. from the University of California, Davis, an M.P.H.
from the University of California, Los Angeles, and a J.D. from Concord
Law School. He is a licensed physician and a member of the State Bar of
California.
Michelle Ann Penny, Ph.D., is a senior director in the translational medicine
group at Eli Lilly and Company. She received her Ph.D. in genetics from
the University of Birmingham, UK, in 1993. After a postdoctoral fellow-
ship in the virology division at the National Institute of Medical Research,
Mill Hill, London, she joined the Imperial College London, Department of
Medical and Community Genetics where she was a postdoctoral research
scientist until taking a lectureship role in human molecular genetics in 1998
as course leader for two master of science programs in human molecular
genetics and molecular genetics with genetic counseling. Her academic
research career focused on the study of complex polygenic diseases, particu-
larly autoimmune disease and susceptibility to infection. In 2002 Dr. Penny
joined the clinical pharmacogenomics group at Pfizer in Sandwich, UK, and
moved to New London, Connecticut, in 2006 to take on the role of oncol-
ogy molecular medicine lead until 2009, when she moved to Indianapolis
to lead the pharmacogenomics work at Eli Lilly and Company.
Aidan Power, M.B., B.Ch., M.Sc., M.R.C.Psych., has been vice president
and head of PharmaTx Precision Medicine since January 2008. Precision
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78 GENOME-BASED THERAPEUTICS
medicine represents a synthesis of all the emerging technologies and opera-
tions (computational science, imaging, pharmacogenomics, m etabolomics,
proteomics, physiological measurements, and diagnostics) that form the
scientific basis of emerging approaches to the development of person-
alized medicine. Graduating in medicine from the University College
Cork, Ireland, Dr. Power trained as a psychiatrist in England and joined
Pfizer in the United Kingdom in 1993, working on the antidepressant
Sertraline and the antipsychotic Ziprasidone. In 2002 Dr. Power relocated
to Pfizer Global Research and Development Headquarters in New London,
Connecticut, where he headed clinical pharmacogenomics. For the last
3 years he has headed up molecular medicine (now PharmaTx Precision
Medicine), which has been integrating molecular studies across disease
areas as well as developing diagnostics for critical programs in the Pfizer
product pipeline.
Sharon Terry, M.A., is president and CEO of the Genetic Alliance, a net-
work dedicated to improving health through the authentic engagement of
communities and individuals. She is the founding CEO of PXE Interna-
tional, a research advocacy organization for the genetic condition pseudo-
xanthoma elasticum (PXE). Following the diagnosis of their two children
with PXE in 1994, Sharon, a former college chaplain, and her husband,
Patrick, founded and built a dynamic organization that enables ethical
research and policies and provides support and information to members
and the public. Along with the other co-inventors of the gene associated
with PXE (ABCC6), she holds the patent for the invention, and with the
assignment of all rights to PXE International, is its steward. She co-directs
a 33-lab research consortium and manages 52 offices worldwide for PXE
International. Ms. Terry is also a co-founder of the Genetic Alliance Reg-
istry and Biobank (GARB). It is a lay-owned and lay-managed biologic
samples and data repository catalyzing translational genomic research on
genetic diseases. GARB works in partnership with academia and industry
to develop novel diagnostics and therapeutics to better understand and
treat these diseases. Ms. Terry is at the forefront of consumer participation
in genetics research, services, and policy and serves as a member of many
of the major governmental advisory committees on biomedical research,
including the Health Information Technology Standards Committee for
the Office of the National Coordinator for Health Information Technol-
ogy, liaison to the Secretary's Advisory Committee on Heritable Disorders
in Newborns and Children and the National Advisory Council for Human
Genome Research, NHGRI, NIH. She serves on the boards of the Institute
of Medicine's Health Sciences Policy Board, National Coalition for Health
Professional Education in Genetics, the Coalition for 21st Century Medi-
cine, and the International Rare Disease Research Consortium. She is on
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APPENDIX B 79
the editorial board of Genetic Testing and Biomarkers, Biopreservation and
Biobanking, and the Google Health and Rosalind Franklin Society Advisory
Boards. Ms. Terry is the chair of the Coalition for Genetic Fairness that was
instrumental in the passage of the Genetic Information Nondiscrimination
Act and is co-chair of the Institute of Medicine's Roundtable on Translat-
ing Genomic-Based Research for Health. In 2005, she received an honor-
ary doctorate from Iona College for her work in community engagement;
the first Patient Service Award from the University of North Carolina
Institute for Pharmacogenomics and Individualized Therapy in 2007; the
Research!America Distinguished Organization Advocacy Award in 2009;
and the Clinical Research Forum and Foundation's Annual Award for
Leadership in Public Advocacy in 2011. She is also an Ashoka Fellow. Ms.
Terry is committed to personal transformation as a catalyst for the system
change needed to improve health and wellness.
Mark Trusheim is a visiting scientist and executive in residence at the
Massachusetts Institute of Technology (MIT) Sloan School of Manage-
ment. He has been a special government employee for the FDA's Office of
the Commissioner and is the founder and president of Co-Bio Consulting,
LLC. He holds degrees in chemistry from Stanford University and manage-
ment from MIT. Mr. Trusheim's research focuses on regional innovation
industry economic clusters and modeling the introduction of new innova-
tions in health care, such as stratified medicines, to inform public policy,
corporate strategy, and product development programs. He is a former
member of the Massachusetts Biotechnology Council's board of directors,
which helps its more than 500 members succeed in the state. In 2004 he
further served as the interim president of the council, leading its success-
ful legislative agenda, its expansion of MassBioEd education programs,
and its continued membership growth. Co-Bio Consulting focuses on bio-
technology public policy, corporate development, and financing. Co-Bio
Consulting helps life sciences firms secure partners and rapidly move their
research to market by connecting strategy formation to action. The firm
also helps facilitate academic, government, and industry consortia to grow
life sciences economic clusters. Clients include established biopharma firms,
start-up biotechs, universities, and government agencies. As an entrepre-
neur, Mr. Trusheim founded and was the first president and chief execu-
tive officer of Cantata Laboratories. Cantata marketed clinical diagnostics
and pharmaceutical biomarker services based on its biochemical profiling
platform. Prior to Cantata, Mr. Trusheim worked at Monsanto/Pharmacia,
culminating his career there as co-president and chief operating officer of
Cereon Genomics, LLC. Located in Cambridge, Massachusetts, Cereon was
created in 1997 by Monsanto as part of a $500 million collaboration with
Millennium Pharmaceuticals. Prior to his position at Cereon, Mr. Trusheim
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80 GENOME-BASED THERAPEUTICS
was responsible for Monsanto's external genomics relationships and held
roles of vice president in the health and wellness sector, marketing director
in Searle Pharmaceutical, and director in the agriculture division strategy.
Mr. Trusheim spent the first half of his career in the high-tech industry
working at Wang Laboratories in computer hardware and at the startup
Kenan Systems Corporation, which focused on developing quantitative
models and artificial intelligencebased applications for large corporations
and government agencies.
